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Using Limited Trial Evidence to Credibly Choose Treatment Dosage when Efficacy and Adverse Effects Weakly Increase with Dose

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  • Charles F. Manski

Abstract

In medical treatment and elsewhere, it has become standard to base treatment intensity (dosage) on evidence in randomized trials. Yet it has been rare to study how outcomes vary with dosage. In trials to obtain drug approval, the norm has been to specify some dose of a new drug and compare it with an established therapy or placebo. Design-based trial analysis views each trial arm as qualitatively different, but it may be highly credible to assume that efficacy and adverse effects (AEs) weakly increase with dosage. Optimization of patient care requires joint attention to both, as well as to treatment cost. This paper develops methodology to credibly use limited trial evidence to choose dosage when efficacy and AEs weakly increase with dose. I suppose that dosage is an integer choice t ∊ (0, 1, . . . , T), T being a specified maximum dose. I study dosage choice when trial evidence on outcomes is available for only K dose levels, where K

Suggested Citation

  • Charles F. Manski, 2023. "Using Limited Trial Evidence to Credibly Choose Treatment Dosage when Efficacy and Adverse Effects Weakly Increase with Dose," NBER Working Papers 31305, National Bureau of Economic Research, Inc.
    • Handle: RePEc:nbr:nberwo:31305
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    JEL classification:

    • C44 - Mathematical and Quantitative Methods - - Econometric and Statistical Methods: Special Topics - - - Operations Research; Statistical Decision Theory
    • I1 - Health, Education, and Welfare - - Health

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